Contemporary Practice and Considerations for Real-World Data Source Identification and Feasibility Assessment.
feasibility
fit‐for‐purpose
real‐world data
real‐world evidence
Journal
Pharmacoepidemiology and drug safety
ISSN: 1099-1557
Titre abrégé: Pharmacoepidemiol Drug Saf
Pays: England
ID NLM: 9208369
Informations de publication
Date de publication:
Sep 2024
Sep 2024
Historique:
revised:
14
06
2024
received:
29
03
2024
accepted:
19
06
2024
medline:
31
8
2024
pubmed:
31
8
2024
entrez:
30
8
2024
Statut:
ppublish
Résumé
There has been rapid growth in the variety and number of real-world data (RWD) sources, as well as the number of regulatory documents that provide guidance for assessing the suitability of RWD sources for pharmacoepidemiology studies. This study aims to assess differences in RWD guidance and variability in current practice for identifying and assessing RWD for studies with regulatory purpose. Key criteria for feasibility assessment were mapped against relevant regulatory guidance documents across US, EU, and Asia-Pacific regions. An online survey was designed and deployed to International Society for Pharmacoepidemiology members to understand current practice. Findings were summarized and used to inform key considerations and recommendations. Eleven RWD guidance documents were identified and mapped against 14 RWD assessment criteria. Variability was seen across these documents in guidance for these criteria. Between December 2022 and January 2023, 37 survey respondents reported having used RWD for post-marketing commitments (34, 92%) and/or background epidemiology (28, 76%). RWD were mostly identified through literature (33, 89%) and data landscaping (26, 70%); guidance documents referenced included: Food and Drug Administration (20, 54%), European Network for Centres for Pharmacoepidemiology and Pharmacovigilance (17, 46%), European Medical Agency (16, 43%), and Structured Process to Identify Fit-For-Purpose Data (11, 30%). Challenges for conducting feasibility assessments included RWD accessibility, ability to complete validation, and RWD provider responsiveness. Existing guidelines are used extensively by researchers, but key criteria for RWD identification and feasibility assessment are not reflected consistently and challenges remain. Recommendations have been made reflecting study findings.
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
e5862Subventions
Organisme : ISPE
Informations de copyright
© 2024 The Author(s). Pharmacoepidemiology and Drug Safety published by John Wiley & Sons Ltd.
Références
U.S. Food & Drug Administration, “Real‐World Data: Assessing Electronic Health Records and Medical Claims Data to Support Regulatory Decision‐Making for Drug and Biological Products, Draft Guidance for Industry,” 2021.
International Council for Harmonisation (ICH), “International Harmonisation of Real‐World Evidence Terminology and Convergence of General Principles Regarding Planning and Reporting of Studies Using Real‐World Data, With a Focus on Effectiveness of Medicines,” 2023, https://admin.ich.org/sites/default/files/2023‐06/ICH_ReflectionPaper_Harmonisation_RWE_Terminology_Endorsed‐ForConsultation_2023_0613.pdf.
A. Makady, A. de Boer, H. Hillege, O. Klungel, and W. Goettsch, “What Is Real‐World Data? A Review of Definitions Based on Literature and Stakeholder Interviews,” Value in Health 20, no. 7 (2017): 858–865.
J. M. Franklin, R. J. Glynn, D. Martin, and S. Schneeweiss, “Evaluating the use of Nonrandomized Real‐World Data Analyses for Regulatory Decision Making,” Clinical Pharmacology & Therapeutics 105, no. 4 (2019): 867–877.
US FDA, “Considerations for the Use of Real‐World Data and Real‐World Evidence to Support Regulatory Decision‐Making for Drug and Biological Products,” 2023, https://www.fda.gov/regulatory‐information/search‐fda‐guidance‐documents/considerations‐use‐real‐world‐data‐and‐real‐world‐evidence‐support‐regulatory‐decision‐making‐drug.
European Network of Centres for Pharmacoepidemiology and Pharmacovigilance (ENCePP), “ENCePP Guide on Methodological Standards in Pharmacoepidemiology,” 2022, http://www.encepp.eu/standards_and_guidances/methodologicalGuide.shtml.
A. Simpson and S. V. Ramagopalan, “R WE Ready for Reimbursement? A Round Up of Developments in Real‐World Evidence Relating to Health Technology Assessment: Part 8,” Journal of Comparative Effectiveness Research 11, no. 13 (2022): 915–917.
European Medicines Agency, “Data Analytics and Methods Task Force. Data Quality Framework for EU Medicines Regulation,” 2022, https://www.ema.europa.eu/en/documents/regulatory‐procedural‐guideline/data‐quality‐framework‐eu‐medicines‐regulation_en.pdf.
M. W. Reynolds, A. Bourke, and N. A. Dreyer, “Considerations When Evaluating Real‐World Data Quality in the Context of Fitness for Purpose,” Pharmacoepidemiology and Drug Safety 29, no. 10 (2020): 1316–1318.
M. Sturkenboom, T. Braeye, L. van der Aa, et al., “Advance Database Characterisation and Fit for Purpose Assessment for Multi‐Country Studies on the Coverage, Benefits and Risks of Pertussis Vaccinations,” Vaccine 38 (2020): B8–B21.
K. M. Duszynski, J. H. Stark, C. Cohet, et al., “Suitability of Databases in the Asia‐Pacific for Collaborative Monitoring of Vaccine Safety,” Pharmacoepidemiology and Drug Safety 30, no. 7 (2021): 843–857.
Center for Drug Evaluation (China), “Center for Drug Evaluation of National Medical Products Administration. Guidelines for Real‐World Study Design and Protocol Framework of Drugs (Draft for Comments)” [in Chinese], 2022, https://www.cde.org.cn/main/news/viewInfoCommon/ea778658adc3d1ae3ffe3f1cc0522e5e.
N. M. Gatto, S. E. Vititoe, E. Rubinstein, R. F. Reynolds, and U. B. Campbell, “A Structured Process to Identify Fit‐for‐Purpose Study Design and Data to Generate Valid and Transparent Real‐World Evidence for Regulatory Uses,” Clinical Pharmacology & Therapeutics 113, no. 6 (2023): 1235–1239.
S. S. McMillan, M. King, and M. P. Tully, “How to use the Nominal Group and Delphi Techniques,” International Journal of Clinical Pharmacy 38 (2016): 655–662.
Pharmaceuticals and Medical Devices Agency, “Points to Consider for Ensuring Data Reliability on Post Marketing Database Study for Drugs,” accessed June 5, 2023, https://www.pmda.go.jp/files/000243761.pdf.
Pharmaceuticals and Medical Devices Agency, “Basic Principles on the Use of Medical Information Databases in Post‐marketing Pharmacovigilance” [translated from Japanese], accessed June 5, 2023, https://www.pmda.go.jp/files/000250561.pdf.
Pharmaceuticals and Medical Devices Agency, “Instructions for Protocols of the Post‐Marketing Database Study” [translated from Japanese], accessed June 5, 2023, https://www.pmda.go.jp/files/000222302.pdf.
M. E. Ritchey and C. J. Girman, “Evaluating the Feasibility of Electronic Health Records and Claims Data Sources for Specific Research Purposes,” accessed October 6, 2022, https://link.springer.com/article/10.1007/s43441‐020‐00139‐x.
ENCePP, “Centres for Pharmacoepidemiology and Pharmacovigilance (ENCePP) Resource Database,” accessed October 2, 2023, https://www.encepp.eu/encepp/resourcesDatabase.jsp. [Note: Centres for Pharmacoepidemiology and Pharmacovigilance (Encepp) Resource Database (Mentioned in Reference 18) Has Been Transferred to EU PAS Register Since the Research].
European Medicines Agency, “List of Metadata for the HMA‐EMA Catalogues of Realworld Data Sources and Studies,” 2022, https://www.ema.europa.eu/en/documents/other/list‐metadata‐real‐world‐data‐catalogues_en.pdf.